Drug Targeting the Lysosome

On November 19-20, 2014, Beyond Batten Disease Foundation (BBDF), together with the New York Academy of Sciences (NYAS), hosted a Drug Targeting the Lysosome Workshop at the New York Academy of Sciences Conference Center at 7 World Trade Center.

This unique think-tank conference featured academic and pharmaceutical researchers who share an interest in designing safe and effective therapies that will target lysosome activity to treat a wide spectrum of human diseases including lysosomal storage diseases like juvenile Batten disease, adult neurodegenerative diseases, and cancer.

Listen to the podcast from the event

Curing Human Diseases: Targeting the Lysosome

Since scientists last met at the “Drug Targeting the Lysosome” conference at the New York Academy of Sciences (NYAS) in New York City in November of 2014. The hard work and preparation of the Scientific Organizing Committee(SOC), lively discussions, and follow-up of attendees has led to published reviews, a podcast for nonscientists, interdisciplinary collaborations funded by Beyond Batten Disease Foundation (BBDF) and the National Institutes of Health (NIH) and a decision to do it all over again.

Immediately following the conference, BBDF and NYAS produced: “Curing Human Diseases: Targeting the Lysosome,” a podcast for nonscientists that can be found on both of our websites and iTunes. Curing Human Diseases explores the potential of lysosomal targeting to treat multiple diseases and why gathering scholars and application scientists from multiple fields will accelerate research and expedite lysosomal targeting’s transition to healthcare.

At the request of Doug Braaten, PhD, SOC members and attendees alike submitted review articles to the Annals of the New York Academy of Sciences. Continuously published since 1823, Annals carries an impact factor of 4.38 and is available to 10,000 institutions worldwide. These articles cover recent discoveries in lysosome biology, the therapeutic potential of drug targeting the lysosome, lysosomal storage disease-relevant cellular and animal models, and specific contributions to disease pathogenesis. Due to the strong interest in these articles, a Special interest issue titled, “Drug Targeting the Lysosome” was created combining these works into a single issue.

Multiple collaborations between attendees resulted in $175,000 in funding from the nonprofit sector and thousands more from the NIH. We are aware of at least 3 laboratories, without previous work in juvenile Batten disease, initiating projects in this area as a result of the conference. Future directions include prize award structures in juvenile Batten disease research and the creation of a consensus document on how to study the lysosome.